Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)

Myasthenia Gravis, Generalized Clinical Trial

Official title:

Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04146051
• Other study ID #: MG-001
• Status: Recruiting
• Phase: Phase 2
• Start date: December 4, 2019
• Est. completion date: March 31, 2026

Study information

• Verified date: May 2024
• Source: Cartesian Therapeutics
• Contact: Study Trial Central Mailbox
• Phone: 302-648-6497
• Email: trials[@]cartesiantx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: March 31, 2026
• Est. primary completion date: March 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patient must be at least 18 years of age.

• Patient must have Generalized Myasthenia Gravis at the time of screening.

• Concomitant immunosuppressive drugs must be deemed necessary by the investigator.

• Seronegative Patients are included

Exclusion Criteria:

• Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient.

• Patient is pregnant or lactating.

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis
• Myasthenia Gravis, Generalized

Intervention

Drug:
• Descartes-08
Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA

Locations

Country	Name	City	State
Canada	University of Alberta	Edmonton	Alberta
United States	Dent Neurologic Institute	Amherst	New York
United States	University of Augusta	Augusta	Georgia
United States	University of Colorado Anschutz Medical Campus	Aurora	Colorado
United States	SFM Clinical Research, LLC	Boca Raton	Florida
United States	Profound Research	Carlsbad	California
United States	University of North Carolina	Chapel Hill	North Carolina
United States	University of California Irvine	Irvine	California
United States	University of Kansas Medical Center Research Institute	Kansas City	Kansas
United States	Neurology Associates, P.A.	Orlando	Florida
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Allegheny Health Network	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Cartesian Therapeutics

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of patients with MG Composite improvement of =5 points.	In Part 3, the primary objective is to compare the effect of Descartes-08 versus placebo, as measured by the change in MGC score from baseline to Week 12. In Part 1, up to three ascending doses of Descartes-08 will be administered to each of 3 to 6 patients; the patients will be staggered at least 21 days apart and will complete a safety review between each dose. In Part 2, patients will receive up to 6 doses in three different schedules depending on the arm they were enrolled into and will be monitored for up to 1 year to assess the safety and tolerability of a repeated dosing schedule.	Day 0 to Day 85
Secondary	Comparison on Descartes-08 versus placebo on QMG and MG ADL	The proportion of patients achieving a =6-point change from Baseline at each posttreatment follow-up in the MG ADL score and QMG score (all parts), and the difference between this proportion in the treatment arm compared to placebo at Day 85.	Day 0 to Day 85
Secondary	Comparison on Descartes-08 versus placebo on MG assessments in crossover patients	The proportion of patients initially randomized to placebo who then cross over to Descartes-08 and achieve a =5-point reduction in MGC score from Crossover Day; 1 to Crossover Day 85.	Day 0 to Day 85
Secondary	Change in titer of myasthenia specific autoantibody titers	Determine the change from baseline over a period of 24 weeks in the titer of myasthenia specific autoantibody titers, e.g. nicotinic acetylcholine receptor autoantibody (anti-nAChR) or muscle-specific tyrosine kinase autoantibody (anti- MusK or anti-Low-Density-Lipoprotein-Related-Protein4 (anti-LRP4) or anti-AChR cluster antibody and the 4 Immunoglobulin types (IgG, IgM, IgA, IgE) following single or multiple infusions of Descartes-08 in MG auto-antibody seropositive patients (Parts 1 and 2 only)	Day 0 to Day 168
Secondary	Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales	Quantify the clinical effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales (MG ADL, QMG, MG QoL 15R, MG PIS, MG Composite) over a period of 24 weeks in MG auto-antibody seropositive and seronegative patients together and separately (all parts).	Day 0 to Day 168

External Links

•   Additional information can be found at Cartformg.org